This is an interesting article from today's WSJ. I think it shows that companies can still be successful even without timely approval from the FDA. The world is a big place and while the US is the most lucrative market if Kevetrin/Purisol/Brilacidin work they will find revenue somewhere...
The following article makes me disdain the FDA even more than I currently do. Like all regulations they are set up to do good but quickly become mired in bureaucracy and focus on their own power, the good of those they are created to protect.
An $8.3 Billion Rebuke to the FDA
Roche buys a drug approved in Europe but not in America.
Aug. 27, 2014 7:12 p.m. ET
Amid this summer's M&A fever, Roche's agreement Monday to buy the San Francisco biotech InterMune ITMN +0.03% deserves special notice. The tie-up is an $8.3 billion guided missile into the fortified bunker that is the Food and Drug Administration.
InterMune has never turned a profit in 16 years of existence and other than its clinical expertise the company holds a single asset: an idea for treating a lethal lung disorder called idiopathic pulmonary fibrosis with no known cause, cure or approved therapy—at least in the U.S. An InterMune drug called pirfenidone that slows the progression of irreversible lung scarring is on the market in Europe, Japan, Canada and even China.
But the FDA refused to approve pirfenidone in 2010, despite the 40,000 Americans who are killed annually by lung fibrosis and a positive recommendation from its outside scientific advisory committee. The agency brass claimed the evidence was statistically unsatisfactory, when one clinical trial was inconclusive but another showed strong benefits such as improved lung function.
The results of the third trial the FDA ordered were reported earlier this year and confirmed that pirfenidone is even more of a treatment advance than it seemed in 2010, and may prolong life. The agency is expected, finally, to approve the medicine in November.
Roche is paying a 38% premium over Friday's closing share price, and 63% over trading before the news of InterMune's corporate suitors broke a few weeks ago. The deal is a big vote of confidence in pirfenidone, not least because a rival lung fibrosis drug is awaiting U.S. approval. Then again, maybe that drug's maker, the German pharmaceutical consortium Boehringer Ingelheim, will have the same FDA experience as InterMune.
The Roche deal is a tacit reprimand to the FDA's unscientific and uncompassionate—and wrong—2010 defenestration. Amid medical ambiguity about effectiveness, the humane option is to allow a drug to come to patients and follow on with more research, in particular for a drug with few side effects. Pulmonary fibrosis is a protracted death sentence of three to five years. The FDA denied tens of thousands of dying people better and possibly longer lives in the time they had left
